AB Science: The clinical development of masitinib in sickle cell
disease is among the 19 winning projects under the sixth call for
“Hospital-Inuversity Research in health (RHU)”
PRESS RELEASE
THE CLINICAL DEVELOPMENT OF MASITINIB IN
SICKLE CELL DISEASE, A HIGHLY PREVALENT GENETIC CONDITION, IS AMONG
THE 19 WINNING PROJECTS UNDER THE SIXTH CALL FOR
"HOSPITAL-UNIVERSITY RESEARCH IN HEALTH (RHU)".
THIS PROGRAM WILL FUND A MASITINIB PHASE
2 CLINICAL TRIAL
AB SCIENCE WILL HOLD A WEBCAST ON THIS
RESEARCH PROGRAM ON THURSDAY, NOVEMBER 30, 2023, FROM 3PM TO 4PM
CET
Paris, November 27, 2023, 8am CET
AB Science SA (Euronext -
FR0010557264 - AB) announces today that a new clinical development
program for masitinib in sickle cell disease is among the 19
winning projects to be funded under the sixth call for
"Hospital-University Research in Health" (Recherche
Hospitalo-Universitaire en santé) projects, which is part of the
Future Investments Program.
The "Hospital-University Research in Health"
(RHU) call for projects of the Future Investments Program, operated
by the National Research Agency, aims to support innovative and
large-scale research projects in the field of health. Focused on
translational research, i.e., converting results in basic research
into results that directly benefit humans, RHU projects involve
academic, hospital, and business stakeholders.
For this sixth wave, an international jury
reviewed 62 applications based on criteria of scientific quality,
innovation, and potential for medical and socio-economic impact. It
proposed to select and fund 19 projects covering various
therapeutic areas and medical needs. These 19 projects will receive
an exceptional grant of 160 million euros from the Future
Investments Program.
As part of this call for projects, the SICKMAST
project, funded with 9.2 million euros, aims to:
-
First, identify and validate, from a database of 1500 patients
(including 700 already identified), biomarkers highlighting the
role of mast cells and basophils in orchestrating acute and chronic
complications of sickle cell disease.
-
Second, demonstrate in a phase 2 clinical trial the efficacy of
masitinib in the treatment of acute and chronic complications of
sickle cell disease in patients identified based on
biomarkers.
The Assistance Publique-Hôpitaux de Paris
(AP-HP) will be the promoter of these phase 2 studies. AB Science
will mainly be involved in supplying masitinib and monitoring
masitinib pharmacovigilance data. AB Science remains free to carry
out, as it sees fit, any potential phase 3 development following
the success of phase 2.
A new patent has been filed, which, if granted,
will extend the international protection of masitinib in sickle
cell disease until 2040. As part of the consortium agreement
established for its patents, AB Science will pay royalties to APHP
in the event of commercialization of masitinib in sickle cell
disease.
Prof. Olivier Hermine, Necker-Enfants malades
AP-HP, coordinator of the SICKMAST program, said, "We are delighted
to be among the laureates of this program, recognized for its
extremely rigorous selection of innovative and medically impactful
projects. Sickle cell disease represents a major public health
challenge due to the number of affected individuals and the early
mortality it causes. This program should allow us to demonstrate
the relevance of selectively targeting two key immune cells in the
treatment of this disease and identifying patients most likely to
respond through new biomarkers."
Alain Moussy, President and co-founder of AB
Science, said, "We are very pleased with this collaboration with
APHP, one of the largest promoters of clinical studies in France,
whose research excellence is globally recognized. This project
demonstrates the full potential of masitinib in targeting certain
key immune cells involved in numerous pathologies. It opens an
extremely promising new development avenue for masitinib and offers
partnership prospects that we will explore to enable the
registration and commercialization of masitinib in this indication
under the best conditions."
A webcast with experts in sickle cell disease
will be held on November 30, 2023, to provide a more detailed
presentation of this project. Login details for this live webcast
will be provided later.
Inflammation mediated by innate immune cells and
promoting vaso-occlusion has recently been shown to play a major
role in sickle cell disease. In particular, our clinical
observations and experimental work in mice, have revealed the
involvement of mast cells and basophils in complications associated
with sickle cell disease:
-
The degree of mast cell activation in patients with sickle cell
disease may contribute to the heterogeneity of inflammation and
chronic and acute complications.
-
The potential role of basophils in sickle cell disease has not been
studied, however, given their role in various diseases and their
ability to release substance P and histamine, they could also play
important roles in the pathophysiology of sickle cell disease.
Masitinib is an inhibitor of KIT, LYN, and FYN,
three major kinases involved in the activation of mast cells and
basophils.
Sickle cell disease (SCD) is an autosomal
recessive disorder affecting millions of people worldwide.
Although life expectancy has increased over the last 20 years,
acute and chronic complications still result in comorbidities, high
social burden and premature death at around 40 years. Approximately
1.1% of couples worldwide are at risk of having a child with a
hemoglobin disorder (sickle cell disease or thalassemia), and 2.3
conceptions per 1,000 are affected by sickle cell disease.
Estimates suggest that each year, around 300,000 children are born
with sickle cell disease, and this number could reach 400,000 by
2050 [1]. Sickle cell disease affects over 100,000 children and
adults in the United States. In France, approximately 26,000
patients are affected (50% children, 50% adults).
The classic view of sickle cell disease
pathophysiology, involves polymerization of mutated hemoglobin
(HbS) leading to red blood cell (RBC) sickling with subsequent
hemolytic anemia, painful vaso-occlusive crisis (VOC) and acute
chest syndrome (ACS).
Current treatment options such as
hydroxycarbamide, chronic transfusion or anti-P-selectin
antibodies, do not fully prevent life-threatening acute and chronic
complications of sickle cell disease. Allogeneic stem cell
transplantation and gene therapy are available only for a minority
of patients, are associated with toxicity and are very expensive,
which limits their use.
There is a significant medical need to prevent
the acute and chronic complications of sickle cell disease.
Reference
[1] Piel FB, Steinberg MH, Rees DC. Sickle Cell
Disease. N Engl J Med. 2017;376(16):1561-1573.
About AB ScienceFounded in
2001, AB Science is a pharmaceutical company specializing in the
research, development and commercialization of protein kinase
inhibitors (PKIs), a class of targeted proteins whose action are
key in signaling pathways within cells. Our programs target only
diseases with high unmet medical needs, often lethal with short
term survival or rare or refractory to previous line of treatment.
AB Science has developed a proprietary portfolio of molecules and
the Company’s lead compound, masitinib, has already been registered
for veterinary medicine and is developed in human medicine in
oncology, neurological diseases, inflammatory diseases and viral
diseases. The company is headquartered in Paris, France, and listed
on Euronext Paris (ticker: AB).
Further information is available on AB Science’s website:
www.ab-science.com.
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